Clinical Trials on Mental Retardation, X-Linked in New Zealand

Total 251 results

Freeline Therapeutics

Completed

AAV Gene Therapy Screening/Observational Protocol (ECLIPSE) (ECLIPSE)

Hemophilia B | Blood Coagulation Disorders, Inherited | Blood Coagulation Disorder | Haematologic Disease

United States, United Kingdom, Australia, Netherlands, Austria, France, Ireland, Italy, Turkey, Canada, Germany, South Africa
Montefiore Medical Center
Eunice Kennedy Shriver National Institute of Child Health and Human Development...

Recruiting

Early Childhood Obesity Programming by Intrauterine Growth Restriction

Childhood Obesity | Epigenetics

United States
Universität Duisburg-Essen
Weinmann Geräte für Medizin GmbH + Co. KG

Completed

Optimum Insufflation Capacity in NMD

Duchenne Muscular Dystrophy | Spinal Muscular Atrophy | Chronic Respiratory Insufficiency

Germany
Shire

Completed

Replagal Enzyme Replacement Therapy for Children With Fabry Disease

Fabry Disease

Canada, United States
Sangamo Therapeutics

Active, not recruiting

Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)

Fabry Disease

United States, United Kingdom, Taiwan, Australia, Germany, Italy, Canada
Novo Nordisk A/S

Completed

Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 in Previously Treated Children With Haemophilia B. (paradigm™5)

Congenital Bleeding Disorder | Haemophilia B

United States, Canada, United Kingdom, France, Malaysia, Brazil, Croatia, Germany, Italy, Japan, Taiwan
Novo Nordisk A/S

Completed

Efficacy of NovoSeven® in Bleeding Prophylaxis in Hemophilia

Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors

France, United States, Spain, Brazil, Philippines, South Africa, Poland, Turkey, Romania, Bulgaria, Russian Federation, Argentina
Mayo Clinic

Recruiting

Prospective Research Rare Kidney Stones (ProRKS) (ProRKS)

Cystinuria | Hyperoxaluria | Dent Disease | Lowe Syndrome | Adenine Phosphoribosyltransferase Deficiency

United States, Canada, Iceland, Israel
Boston Children's Hospital
Massachusetts General Hospital; Mayo Clinic; Karolinska University Hospital; Baylor... and other collaborators

Active, not recruiting

Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita

Aplastic Anemia | Dyskeratosis Congenita | Hoyeraal Hreidarsson Syndrome | Revesz Syndrome

United States, Norway, Sweden
Amicus Therapeutics

Terminated

Open-Label Phase 3 Long-Term Safety Study of Migalastat (AT1001-041)

Fabry Disease

Australia, United States, Italy, Spain, Belgium, Turkey, Brazil, Argentina, United Kingdom, Denmark, France, Canada, Egypt
Amicus Therapeutics

Completed

Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease

Fabry Disease

United States, Austria, United Kingdom, Argentina, Australia, Belgium, Brazil, Canada, Denmark, Egypt, France, Italy, Japan, Spain, Turkey
Genzyme, a Sanofi Company

Recruiting

Fabry Disease Registry & Pregnancy Sub-registry

Fabry Disease

Croatia, United States, Argentina, Australia, Belgium, Bulgaria, Canada, Germany, Hong Kong, India, Indonesia, Korea, Republic of, Kuwait, Malaysia, Philippines, Poland, Romania, Singapore, Slovakia, Taiwan, Thailand, United Kingdom, Vietn... and more
The University of Queensland

Recruiting

The TELO-SCOPE Study: Attenuating Telomere Attrition With Danazol. Is There Scope to Dramatically Improve Health Outcomes for Adults and Children With Pulmonary Fibrosis

Pulmonary Fibrosis | Dyskeratosis Congenita | Telomere Shortening | Telomere Disease

Australia
Novo Nordisk A/S

Completed

Efficacy and Safety of NNC-0156-0000-0009 During Surgical Procedures in Subjects With Haemophilia B (paradigm™ 3)

Congenital Bleeding Disorder | Haemophilia B

United States, Netherlands, Austria, Spain, Germany, Macedonia, The Former Yugoslav Republic of, Taiwan, United Kingdom, Italy, Malaysia, Thailand, France, Greece, Turkey, Japan, South Africa, Romania
Novo Nordisk A/S

Completed

Safety and Efficacy of NNC-0156-0000-0009 in Haemophilia B Patients (paradigm™ 2)

Congenital Bleeding Disorder | Haemophilia B

United States, Netherlands, Germany, Macedonia, The Former Yugoslav Republic of, United Kingdom, Italy, Malaysia, Hungary, Thailand, France, Russian Federation, Turkey, Japan, South Africa, Canada
Amicus Therapeutics

Completed

Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease

Fabry Disease

United States, Italy, Spain, Turkey, Brazil, Canada, United Kingdom, France, Poland, Australia, Egypt, Argentina, Denmark
Ultragenyx Pharmaceutical Inc
Kyowa Kirin Co., Ltd.

Active, not recruiting

X-linked Hypophosphatemia Disease Monitoring Program

X-linked Hypophosphatemia | Hypophosphatemic Rickets

United States, Brazil, Argentina, Canada, Chile, Colombia
Genzyme, a Sanofi Company

Completed

A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

Fabry Disease

United States, United Kingdom, Canada, Czech Republic, Hungary, Poland
Novo Nordisk A/S

Completed

PROPACT: Retrospective Prophylaxis Patient Case Collection (PROPACT)

Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors

Slovakia, Spain, Argentina, Germany, Croatia, Italy, Canada, United Kingdom, Ireland, Sweden, France, United States, Czech Republic, Switzerland
Spark Therapeutics

Completed

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors

Hematologic Diseases | Blood Coagulation Disorders, Inherited | Coagulation Protein Disorders | Hemorrhagic Disorders | Genetic Diseases, Inborn | Genetic Diseases, X-Linked | Gene Therapy | Blood Coagulation Disorder | Gene Transfer | Adeno-Associated Virus (AAV) | Factor VIII (FVIII) | Factor VIII (FVIII)... and other conditions

United States
Mendel Tuchman
Children's Hospital of Philadelphia; University of California, Los Angeles; Icahn... and other collaborators

Completed

Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia (STO)

Methylmalonic Acidemia | Carbamoyl-Phosphate Synthase I Deficiency Disease | Propionic Acidemia, Type I and/or Type II | Ornithine Carbamoyltransferase Deficiency

United States
Novo Nordisk A/S

Completed

Haemophilia Patients With Inhibitors Being Treated for Acute Joint Bleeds

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

United States, Spain, Taiwan, Turkey, Poland, Croatia, Italy, Malaysia, Brazil, United Kingdom, Hungary, Israel, Thailand, Japan, South Africa, Canada, France, Argentina
Genzyme, a Sanofi Company

Completed

A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease

Fabry Disease

United States, United Kingdom, Canada, Hungary, Poland, Czech Republic
Novo Nordisk A/S

Completed

Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B (paradigm™6)

Congenital Bleeding Disorder | Haemophilia B

Spain, United Kingdom, Australia, Taiwan, France, Malaysia, United States, Austria, Canada, Israel, Japan, Thailand, Algeria, Argentina
Columbia University

Completed

Impact of Cyanoacrylate Glue on PICC Line Dressing Care

Central Line Complication | Neonatal Disease

United States
Sanofi

Recruiting

A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease (CARAT)

Fabry Disease

Italy, United States, Austria, China, Czechia, Denmark, France, Germany, Greece, Japan, Korea, Republic of, Netherlands, Norway, Spain, Taiwan, Turkey, United Kingdom, Canada, Poland
Novo Nordisk A/S

Completed

Efficacy and Safety of NNC 0078-0000-0007 in Patients With Congenital Haemophilia and Inhibitors (adept™2)

Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors

Taiwan, United Kingdom, Thailand, Serbia, Croatia, Italy, Poland, Romania, Hungary, Malaysia, United States, Austria, Brazil, Greece, Japan, Puerto Rico, Russian Federation, South Africa, Turkey
Grifols Therapeutics LLC

Completed

Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency Patients

Common Variable Immunodeficiency | Severe Combined Immunodeficiency | Wiskott-Aldrich Syndrome | Agammaglobulinemia | Immunologic Deficiency Syndrome

Canada, United States
Sanofi

Recruiting

A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease (PERIDOT)

Fabry Disease

China, Finland, Germany, Norway, United Kingdom, United States, Denmark, Austria, Greece, Romania, Argentina, Brazil, Canada, Japan, Mexico, Poland, Italy, France, Turkey, Australia, Switzerland
National Taipei University of Nursing and Health...

Active, not recruiting

Acupuncture-like Transcutaneous Electrical Nerve Stimulation on Bowel Symptom and Quality of Life in Patients With Irritable Bowel Syndrome

Irritable Bowel Syndrome | Acupuncture-like Transcutaneous Electrical Nerve Stimulation

Taiwan
Fundació Institut de Recerca de l'Hospital de la...
Fundacion Clinic per a la Recerca Biomédica

Recruiting

sFlt1/PlGF and Planned Delivery to Prevent Preeclampsia at Term. (PE37)

Preeclampsia | Intrauterine Growth Restriction | Perinatal Death | Maternal Hypertension | Neonatal Outcome

Spain, Belgium, Czechia, Poland, India
Medical College of Wisconsin
National Cancer Institute (NCI); National Heart, Lung, and Blood Institute... and other collaborators

Completed

Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204) (RICHI)

Chronic Granulomatous Disease | Hemophagocytic Lymphohistiocytosis | Leukocyte Adhesion Deficiency | Chronic Active Epstein-Barr Virus Infection | HIGM-1 | IPEX

United States, Canada
Sanofi

Terminated

Atherosclerosis Underlying Development Assessed by Intima-Media Thickness in Patients on Rimonabant (AUDITOR)

Arteriosclerosis | Obesity | Carotid Artery Plaque | Metabolic Syndrome X

United States, France, Canada, Netherlands, Spain, United Kingdom
American Thrombosis and Hemostasis Network
Sanofi; Pfizer; Genentech, Inc.; CSL Behring; Hemophilia of Georgia, Inc.; Hemab Therapeutics

Recruiting

ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders

Hemophilia | Thrombosis | Thrombophilia | Sickle Cell Disease | Thalassemia | Bleeding Disorder | Von Willebrand Diseases | Factor IX Deficiency | Connective Tissue Disorder | Factor VIII Deficiency | Hematologic Disorder | Rare Bleeding Disorder | Platelet Disorder

United States
Natera, Inc.
Children's Hospital of Philadelphia; Montefiore Medical Center; University of... and other collaborators

Completed

SNP-based Microdeletion and Aneuploidy RegisTry (SMART) (SMART)

Prader-Willi Syndrome | DiGeorge Syndrome | 22q11 Deletion Syndrome | Angelman Syndrome | Trisomy 21 | Trisomy 18 | Trisomy 13 | Monosomy X | Sex Chromosome Abnormalities | 1p36 Deletion Syndrome | Cri-du-Chat Syndrome

United States, Australia, Ireland, Spain, Sweden, United Kingdom
Novartis Pharmaceuticals

Completed

A Phase II Study to Evaluate the Efficacy of TKI258 for the Treatment of Patients With FGFR2 Mutated or Wild-type Advanced and/or Metastatic Endometrial Cancer

Endometrial Cancer | VEGF | Solid Tumors and Advanced Endometrial Cancer | Second-line Treatment

Italy, United Kingdom, Spain, Brazil, United States, Korea, Republic of, New Zealand
Bristol-Myers Squibb

Completed

Phase II Study of Ipilimumab Monotherapy in Recurrent Platinum-sensitive Ovarian Cancer

Platinum-sensitive Ovarian Cancer, Second-line, Third-line, or Fourth-line

United States
Fraser Health
SterileCare Inc.; Center for Health Evaluation & Outcome Services

Recruiting

CLiCK in the Critical Care Unit (CLiCK)

Central Venous Catheter Thrombosis | Catheter Infection | Catheter Complications | Catheter | Catheter Blockage | Catheter Dysfunction | Central Line-associated Bloodstream Infection (CLABSI) | Central Venous Catheter Related Bloodstream Infection

Canada
Mirvie

Active, not recruiting

MIRACLE of LIFE Study (MoL)

Preterm Birth | Preterm Labor | Preeclampsia | Intrauterine Growth Restriction | Gestational Diabetes Mellitus in Pregnancy | Small for Gestational Age at Delivery | Preterm Birth Complication | HELLP | Preterm Premature Rupture of Membrane | Gestational Hypertension

United States
National Cancer Institute (NCI)

Active, not recruiting

Combination Chemotherapy in Treating Young Patients With Newly Diagnosed High-Risk B Acute Lymphoblastic Leukemia and Ph-Like TKI Sensitive Mutations

B Acute Lymphoblastic Leukemia | Central Nervous System Leukemia | Testicular Leukemia | B Acute Lymphoblastic Leukemia, BCR-ABL1-Like

United States, Canada, Puerto Rico, Australia, New Zealand, Switzerland, Ireland
Hereditary Neuropathy Foundation

Recruiting

Natural History Study for Charcot Marie Tooth Disease

Charcot-Marie-Tooth Disease | Charcot-Marie-Tooth Disease, Type IA | Charcot-Marie-Tooth Disease Type 2A | Charcot-Marie-Tooth | Charcot-Marie-Tooth Disease, Type IB | Charcot-Marie-Tooth Disease Type 2 | Charcot-Marie-Tooth Disease, Type 2C | Charcot-Marie-Tooth Disease Type 2A2B | Charcot-Marie-Tooth... and other conditions

United States
Roswell Park Cancer Institute

Active, not recruiting

Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer

Acute Myeloid Leukemia | Polycythemia Vera | Myelofibrosis | Chronic Myelomonocytic Leukemia | Waldenstrom Macroglobulinemia | Acute Lymphoblastic Leukemia | Chronic Lymphocytic Leukemia | Secondary Acute Myeloid Leukemia | Sickle Cell Disease | Myelodysplastic Syndrome | Plasma Cell Myeloma | Chronic Granulomatous... and other conditions

United States
Dren Bio
Novotech

Recruiting

A Study of DR-01 in Subjects With Large Granular Lymphocytic Leukemia or Cytotoxic Lymphomas

Aggressive NK Cell Leukemia | Hepatosplenic T-cell Lymphoma | Enteropathy-Associated T-Cell Lymphoma | Subcutaneous Panniculitis-Like T-Cell Lymphoma | Monomorphic Epitheliotropic Intestinal T-Cell Lymphoma | LGLL - Large Granular Lymphocytic Leukemia | Primary Cutaneous T-Cell Lymphoma - Category and other conditions

United States, Australia, France, Spain
Jonathann Kuo, MD

Active, not recruiting

Dual Sympathetic Blocks for Patients Experiencing Sympathetically-Mediated Symptoms From Long COVID (DSBLongCOVID)

SARS-CoV2 Infection | Post-COVID-19 Syndrome | Dysautonomia | Post Acute COVID-19 Syndrome | Long COVID | Long Covid19 | COVID-19 Recurrent | Post-Acute COVID-19 | Post-Acute COVID-19 Infection | Post Acute Sequelae of COVID-19 | Dysautonomia Like Disorder | Dysautonomia Orthostatic Hypotension Syndrome | Post... and other conditions

United States
National Cancer Institute (NCI)

Completed

Veliparib in Treating Patients With Malignant Solid Tumors That Do Not Respond to Previous Therapy

Recurrent Fallopian Tube Carcinoma | Recurrent Ovarian Carcinoma | Recurrent Primary Peritoneal Carcinoma | Breast Carcinoma | Ovarian Carcinoma | Pancreatic Carcinoma | Prostate Carcinoma | Recurrent Breast Carcinoma | Estrogen Receptor Negative | HER2/Neu Negative | Progesterone Receptor Negative | Triple-Negative... and other conditions

United States
Tisento Therapeutics
Children's Hospital of Philadelphia

Terminated

Phase 2a Study of IW-6463 in Adults Diagnosed With Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS)

MELAS

United States
Tisento Therapeutics

Not yet recruiting

A Phase 2b Study of Zagociguat in Patients With MELAS (PRIZM)

Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)

United States, Australia, Italy, Germany, Canada, United Kingdom
AstraZeneca
Merck Sharp & Dohme LLC; GOG Foundation; Myriad Genetic Laboratories, Inc.

Active, not recruiting

Olaparib Maintenance Monotherapy in Patients With BRCA Mutated Ovarian Cancer Following First Line Platinum Based Chemotherapy. (SOLO-1)

Advanced Ovarian Cancer | Newly Diagnosed | FIGO Stage III-IV | Partial Response | Complete Response | BRCA Mutation | First Line Platinum Chemotherapy

Korea, Republic of, France, Italy, United States, Israel, Canada, China, Brazil, United Kingdom, Poland, Australia, Japan, Netherlands, Russian Federation, Spain
Roswell Park Cancer Institute

Completed

Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies

Primary Myelofibrosis | Polycythemia Vera | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma and other conditions

United States
Memorial Sloan Kettering Cancer Center

Recruiting

A Registry for People With T-cell Lymphoma

Mycosis Fungoides | Sezary Syndrome | Angioimmunoblastic T-cell Lymphoma | Hepatosplenic T-cell Lymphoma | Anaplastic Large Cell Lymphoma, ALK-Positive | Extranodal NK/T-cell Lymphoma, Nasal Type | T-cell Lymphoma | Peripheral T-Cell Lymphoma, Not Otherwise Specified | Primary Cutaneous Anaplastic Large... and other conditions

United States

Clinical Trials on Mental Retardation, X-Linked in New Zealand

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Turkmenistan

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations